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U.K. Clears Glaxo's $700,000 ‘Bubble Boy Disease’ Gene Therapy

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  • Health authority approves country’s most expensive medicine
  • Only 4 patients treated with gene therapy Strimvelis so far

The U.K.’s thrifty health authority has cleared its most expensive medicine ever -- a $700,000 GlaxoSmithKline Plc treatment for an illness known as “bubble-boy disease.” But the overall cost to the country’s medical system won’t be as eye-popping as the price suggests.

Glaxo, Britain’s biggest drugmaker, said by email it has treated four patients with Strimvelis since it was approved by the European Union in May 2016. The one-time procedure and potential cure, targeting an ultra-rare disease that leaves newborn babies unable to fight off everyday infections, is priced at 594,000 euros ($716,000).

Strimvelis, only the second gene therapy for an inherited disease to be licensed anywhere in the world, was recommended Wednesday by the U.K.’s National Institute for Health and Care Excellence, which determines whether new drugs are cost-effective.

While gene therapies are revolutionizing treatment for an array of diseases after overcoming decades of challenges, questions remain about their commercial prospects and pricing. Spark Therapeutics Inc. last month won U.S. approval of its gene therapy for an inherited form of blindness. Since the therapy is intended to be administered only once, Spark said it will charge $850,000 for both eyes.

One Hospital

Glaxo shares rose 1.9 percent to 1,347 pence at 1:58 p.m. in London trading. The company says two more patients are due to be treated soon with Strimvelis, currently available only at a hospital in Milan. The patient numbers reflect the rare incidence of the disease, a spokesman for the London-based company said in an email. The treatment is not available in the U.S.

About three babies a year in England are born with the condition, called severe combined immunodeficiency syndrome due to adenosine deaminase deficiency, or ADA-SCID. It’s informally known as “bubble-boy disease” after a 1976 film starring John Travolta about a patient living in a sterile environment.

While stem-cell transplants, the current treatment, can restore the immune system, they may not succeed in all cases, and closely matched donors are hard to find or may not be available, according to the U.K. drug-price monitor.

Real Cost

The cost of drugs has been a contentious issue in the U.K. with the health adviser objecting to paying for some new medicines, saying their benefits don’t justify their cost. The country last year introduced new funding limits, prompting opposition from drugmakers.

The list price of Strimvelis would make it the most expensive medicine ever recommended in the U.K., according to the agency. Yet the estimated net budget impact to the system would be 2.35 million pounds ($3.2 million) over five years, according to documents. By contrast, about one in five therapies approved in the country cost more than 20 million pounds a year, including treatments for hepatitis C, NICE estimated last year.

Glaxo last year signaled it may consider a sale of its rare diseases unit, which includes Strimvelis. A spokesman said in November that the company is seeking the “right partner who can ensure these medicines get the focus they deserve and are available to the patients that need them.”

The therapy is recommended when a suitable stem cell donor cannot be found. Organizations consulted on the therapy have an opportunity to appeal.

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